Abeona Therapeutics
NASDAQ:ABEOAbeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.
Aquestive Therapeutics
NASDAQ:AQSTAquestive Therapeutics, Inc. operates as a pharmaceutical company in the United States and internationally. The company markets Sympazan, an oral soluble film formulation of clobazam for the treatment of lennox-gastaut syndrome; Suboxone, a sublingual film formulation of buprenorphine and naloxone for the treatment of opioid dependence; Zuplenz, an oral soluble film formulation of ondansetron for the treatment of nausea and vomiting associated with chemotherapy and post-operative recovery; and Azstarys, a once-daily product for the treatment of attention deficit hyperactivity disorder. Its proprietary product candidates comprise Libervant, a buccal soluble film formulation of diazepam for the treatment of seizures; KYNMOBI, a sublingual film formulation of apomorphine for the treatment of episodic off-periods in Parkinson's disease; and Exservan, an oral soluble film formulation of riluzole for the treatment of amyotrophic lateral sclerosis. The company's proprietary pipeline of complex molecule product includes AQST-108, a sublingual film formulation delivering systemic epinephrine for the treatment of conditions other than anaphylaxis; and Anaphylm, an epinephrine sublingual film for the emergency treatment of allergic reactions, including anaphylaxis. In addition, it develops Adrenaverse, an epinephrine prodrug platform. The company was incorporated in 2004 and is headquartered in Warren, New Jersey.
Correvio Pharma
NASDAQ:CORVCorrevio Pharma Corp., a specialty pharmaceutical company, engages in developing therapeutics worldwide. Its portfolio of marketed brands comprise Aggrastat (tirofiban hydrochloride) for patients with acute coronary syndrome; and Brinavess (vernakalant IV) for the rapid conversion of recent onset atrial fibrillation to sinus rhythm. The company also offers ESMOCARD and ESMOCARD LYO, a short acting beta blocker for the treatment of supraventricular tachycardia and control of the ventricular rate in patients with atrial fibrillation; Trevyent, a development stage drug/device combination product for pulmonary arterial hypertension; Xydalba, a treatment for acute bacterial skin and skin structure infections; and Zevtera/Mabelio, a cephalosporin antibiotic for intravenous administration with rapid bactericidal activity. Correvio Pharma Corp. was incorporated in 2018 and is headquartered in Vancouver, Canada.
Eton Pharmaceuticals
NASDAQ:ETONEton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.
Verastem
NASDAQ:VSTMVerastem, Inc., a development-stage biopharmaceutical company, focuses on developing and commercializing drugs for the treatment of cancer in the United States. Its product candidates are Avutometinib, an orally available small molecule RAF/MEK clamp that inhibits the ras sarcoma RAF/MEK, ERK mitogen activated pathway kinase pathway which is involved in cell proliferation, migration, transformation, and survival of tumor cells; and Defactinib, an oral small molecule inhibitor of FAK and proline-rich tyrosine kinase for various solid tumors. The company is involved in clinical studies, including RAMP 301, a randomized global confirmatory trial to evaluate the combination of Avutometinib and Defactinib for the treatment of patients with recurrent low-grade serous ovarian cancer; RAMP 201, an adaptive two-part multicenter, parallel cohort, randomized open label trial to evaluate the efficacy and safety of Avutometinib and in combination with Defactinib; and FRAME, an investigation of Avutometinib and Defactinib in patients with KRAS mutant cancers and subsequent analyses; and RAMP 204 and 205. It has license agreements with Chugai Pharmaceutical Co., Ltd. for the development, commercialization, and manufacture of products containing Avutometinib; and Pfizer Inc. to research, develop, manufacture, and commercialize products containing Pfizer's inhibitors of FAK for therapeutic, diagnostic, and prophylactic uses in humans. In addition, it has a clinical collaboration agreement with Amgen, Inc. to evaluate the combination of Avutometinib with Amgen's KRAS-G12C inhibitor LUMAKRAS which in Phase 1/2 trial entitled RAMP 203; and a discovery and development collaboration with GenFleet Therapeutics to advance new programs targeting RAS pathway-driven cancers. Verastem, Inc. was incorporated in 2010 and is headquartered in Needham, Massachusetts.